Core Viewpoint - uniQure N.V. (QURE) experienced a significant stock surge of 109.7% following the announcement of alignment with the FDA on an accelerated approval pathway for AMT-130, a gene therapy aimed at treating Huntington's disease [1] Company Developments - The FDA's Regenerative Medicine Advanced Therapy (RMAT) designation has been granted to AMT-130, which is designed to treat Huntington's disease [2] - An RMAT meeting in November 2024 confirmed that existing data from early to mid-stage studies of AMT-130 can serve as the primary basis for a regulatory filing, eliminating the need for an additional pre-submission study [3] - The FDA has accepted the use of cUHDRS as an intermediate clinical endpoint and reductions in neurofilament light (NfL) in cerebrospinal fluid (CSF) as supportive evidence for therapeutic benefit [4] - uniQure is preparing to submit a biologics license application to the FDA for AMT-130 and plans to engage in further discussions regarding statistical analysis and technical requirements in the first half of 2025 [5] Clinical Data and Efficacy - Updated interim data from July 2024 indicated a potential long-term clinical benefit and reduction of neurodegeneration markers in patients treated with AMT-130 [6][7] - Two phase I/II studies are being conducted, with 29 patients treated and a total of 21 patients having 24-month follow-up data available [8] - The treatment has shown a manageable safety profile, with no new drug-related serious adverse events reported [9] Future Plans and Pipeline - uniQure aims to complete patient enrollment in the third cohort of the U.S. phase I/II study soon, with safety data expected in the first half of 2025 [10] - An interim analysis comparing treated patients to external controls is planned for mid-2025 [11] - The company has additional candidates in early-stage development for conditions such as refractory temporal lobe epilepsy, amyotrophic lateral sclerosis, and Fabry disease [12] Market Position - uniQure markets Hemgenix, a gene therapy for hemophilia B, which received approvals in the U.S. and EU in 2022 and 2023, respectively [13] - The company currently holds a Zacks Rank 2 (Buy), indicating a favorable market position [14]

Core Viewpoint - Shares of uniQure (QURE) have increased by 5.2% recently, closing at $6.02, with a potential upside indicated by Wall Street analysts' price targets suggesting a mean estimate of $19.75, representing a 228.1% upside [1] Price Targets - The average of 12 short-term price targets ranges from a low of $7 to a high of $28, with a standard deviation of $8.05, indicating variability among estimates [2] - The lowest estimate suggests a 16.3% increase from the current price, while the highest estimate indicates a 365.1% upside [2] - A low standard deviation signifies greater agreement among analysts regarding price movement [2][7] Analyst Sentiment - Analysts show increasing optimism about QURE's earnings prospects, as evidenced by a strong consensus in revising EPS estimates higher, which correlates with potential stock price increases [4][9] - Over the past 30 days, five estimates have been revised upward, resulting in a 7.1% increase in the Zacks Consensus Estimate for the current year [10] Zacks Rank - QURE holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate factors, indicating strong potential for upside [11] Conclusion on Price Targets - While consensus price targets may not reliably indicate the extent of potential gains, they can provide a directional guide for price movement [12]

uniQure (QURE) could be a solid choice for investors given its recent upgrade to a Zacks Rank #2 (Buy). This upgrade primarily reflects an upward trend in earnings estimates, which is one of the most powerful forces impacting stock prices.The sole determinant of the Zacks rating is a company's changing earnings picture. The Zacks Consensus Estimate -- the consensus of EPS estimates from the sell-side analysts covering the stock -- for the current and following years is tracked by the system.Since a changing ...

uniQure (QURE) closed the last trading session at $7.43, gaining 37.1% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $20 indicates a 169.2% upside potential.The average comprises 12 short-term price targets ranging from a low of $7 to a high of $28, with a standard deviation of $7.82. While the lowest estimate indicates a decline of 5.8% from the current price level, the most opt ...

UniQure (QURE) came out with a quarterly loss of $0.91 per share versus the Zacks Consensus Estimate of a loss of $1.12. This compares to loss of $1.88 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 18.75%. A quarter ago, it was expected that this human gene therapy company would post a loss of $1.24 per share when it actually produced a loss of $1.16, delivering a surprise of 6.45%.Over the last four quarters, the company ha ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _______ to _______ Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands ...

Exhibit 99.1 uniQure Announces Third Quarter 2024 Financial Results and Highlights Recent Company Progress ~ Type B meeting scheduled with the FDA in the fourth quarter of 2024 to initiate discussions regarding a potential expedited clinical development pathway for AMT-130 in Huntington's disease ~ ~ Presented positive interim data from Phase I/II trial of AMT-130 demonstrating slowing of Huntington's disease progression and reductions in key biomarker of neurodegeneration ~ ~ Initiated patient dosing in ne ...

~ Type B meeting scheduled with the FDA in the fourth quarter of 2024 to initiate discussions regarding a potential expedited clinical development pathway for AMT-130 in Huntington’s disease ~ ~ Presented positive interim data from Phase I/II trial of AMT-130 demonstrating slowing of Huntington’s disease progression and reductions in key biomarker of neurodegeneration ~ ~ Initiated patient dosing in new Phase I/II studies of AMT-162 in SOD1-ALS and AMT-191 in Fabry disease; First patient enrolled in observa ...

UniQure (QURE) came out with a quarterly loss of $1.16 per share versus the Zacks Consensus Estimate of a loss of $1.24. This compares to loss of $1.44 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of 6.45%. A quarter ago, it was expected that this human gene therapy company would post a loss of $1.31 per share when it actually produced a loss of $1.36, delivering a surprise of -3.82%. Over the last four quarters, the company ...

Exhibit 99.1 uniQure Announces Second Quarter 2024 Financial Results and Provides Company Update ~ Announced RMAT designation for AMT-130 in Huntington's disease and positive interim Phase I/II data demonstrating the slowing of disease progression and reductions in a key biomarker of neurodegeneration; Meeting with FDA expected in the second half of 2024 to discuss potential for expedited clinical development ~ ~ Initiated patient screening for three additional Phase I/II studies in mesial temporal lobe epi ...